The US National Institute of Health (NIH) has announced plans to provide funding for three clinical trials to investigate drugs for the treatment of amyotrophic lateral sclerosis (ALS).

The three trials follow the 2021 implementation of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS). ACT for ALS calls for the establishment of a grant programme for research using data from expanded access programmes (EAPs) to access investigational drugs for individuals who are not otherwise eligible for ALS-related clinical trials.

The first trial (U01NS136021) will be led by clinical-stage biotech company Prilenia Therapeutics and researchers at Massachusetts General Hospital. The trial will investigate pridopidine, a small molecule drug that activates the Sigma-1 receptor, which is found on neurons and has been linked to ALS in genetic and preclinical studies.

The study intends to enrol 200 people across 45 clinical sites in the US
The second trial (U01NS136023) will be conducted by Clene Nanomedicine and Columbia University and investigate the safety and efficacy of CNM-Au8 in 100 patients across 10 clinical sites and a virtual clinic by Synapticure. This trial was awarded $45.1m by the National Institute of Neurological Disorders and Stroke (NINDS), a sub-division of the NIH.

The study will examine CNM-Au8, an orally administered suspension made of gold nanocrystals that may protect neurons by raising levels of energy molecules in cells.
Clene’s head of medical Benjamin Greenberg detailed how Clene has demonstrated evidence of consistent safety and improved survival for CNM-Au8 across a broad ALS population in two independent Phase II trials and an ongoing EAP with up more than three and a half years of follow-up.

Greenberg said: “This new EAP provides access to CNM-Au8 for more people living with ALS and enables the collection of survival, safety and biomarker data in a population not studied in clinical trials. These data can help provide confirmatory support for the existing trial data Clene has gathered in its clinical trials.”

The final trial (U01NS136020) will be led by Rapa Therapeutics and researchers at Massachusetts General Hospital. It will investigate RAPA-501, a cell therapy that is designed to reduce inflammation, which has the potential to slow disease progression.

The trial will enrol 40 people across seven clinical sites to look at immune system activity. The study will use standard methods and machine-learning-based algorithms to examine treatment efficacy.

According to GlobalData’s database, there are 39 neuroimmunology drugs in Phase I, II, and III stages of development for the treatment of ALS. In the wider field of Neuroimmunology, the sale of relevant drugs is expected sales is expected to increase from $20.1bn in 2020 to $26.6bn by 2025.

GlobalData is the parent company of Clinical Trials Arena.